What is Yargesa and Miglustat used for?
Yargesa is a medicine used to treat type 1 Gaucher disease in adults with mild to moderate symptoms.
Patients suffering from this disease show the lack of an enzyme that degrades a type of fat called glucosylceramide. Consequently, glucosylceramide accumulates in different parts of the body, such as the spleen, liver and bones. Yargesa is used in patients who cannot be subjected to enzyme replacement therapy.
Yargesa is a "generic medicine". This means that it contains the same active substance (miglustat) and acts in the same way as a "reference medicine" already authorized in the European Union (EU) called Zavesca. For more information on generic medicines, see the questions and answers by clicking here.
How is Yargesa - Miglustat used?
Yargesa is available in 100 mg capsules to be taken orally. The recommended starting dose is one capsule three times a day. A lower dose should be used in patients with renal impairment and those who develop diarrhea.
The medicine can only be obtained with a prescription and treatment must be monitored by physicians experienced in the management of Gaucher disease.
For more information, see the package leaflet.
How does Yargesa - Miglustat work?
The active substance in Yargesa, miglustat, inhibits the action of an enzyme called glucosylceramide synthase. This enzyme is involved in the first phase of glucosylceramide production. By preventing the enzyme from acting, miglustat can reduce the production of glucosylceramide in cells and thus alleviate the symptoms of type 1 Gaucher disease.
What benefits has Yargesa - Miglustat shown during the studies?
Studies on the benefits and risks of the active ingredient for the approved use have already been carried out with the reference medicinal product (Zavesca) and must therefore not be repeated for Yargesa.
As with any medicine, the company has made studies on the quality of Yargesa available. In addition, he performed a study that showed his "bioequivalence" with the reference medicine. Two medicines are bioequivalent when they produce the same levels of active ingredient in the body, so they are expected to have the same effect.
What are the risks associated with Yargesa - Miglustat?
Because Yargesa is a generic medicine and is bioequivalent to the reference medicine, its benefits and risks are considered to be the same as those of the reference medicine.
Why has Yargesa - Miglustat been approved?
The Agency's Committee for Medicinal Products for Human Use (CHMP) concluded that, in accordance with EU requirements, Argesa has been shown to have comparable quality and to be bioequivalent to Zavesca. Therefore, the CHMP considered that, as in the case of Zavesca, the benefits outweigh the identified risks and recommended to approve the use of EU law.
What measures are being taken to ensure the safe and effective use of Yargesa - Miglustat?
Recommendations and precautions to be followed by healthcare professionals and patients for the safe and effective use of Yargesa have been included in the summary of product characteristics and the package leaflet.
More information on Yargesa - Miglustat
For the full EPAR for Yargesa consult the Agency's website: ema.europa.eu/Find medicine / Human medicines / European public assessment reports. For more information on Yargesa therapy, read the package leaflet (also part of the EPAR) or contact your doctor or pharmacist.
The full EPAR for the reference medicine can also be found on the Agency's website.
